- According to the authors of a new study published in the journal Therapeutic Innovation and Regulatory Science, the FDA has approached orphan drug products "with a meaningful and reasonable flexibility," RAPS reports.
- The 1983 Orphan Drug Act defines a rare disease as a disease that affects fewer than 200,000 patients in the U.S. Under this act, drugs approved under this provision are eligible for a seven-year period of market exclusivity, as well as tax credits, grants and user fee waivers.
- However, this does not mean that these drug makers are given an expedited review process.
According to advocates, there are no approved treatments for 95% of the 7,000 orphan diseases. Nonetheless, the Orphan Drug Act has been a true game-changer. Prior to the passage of this act, there were only a few orphan drug approvals each year; however since passage of the act that number has increased substantially, with 49 approvals in 2014.
Although orphan drugs are subject to the same regulatory rigor as other drugs, the FDA has been found to be flexible with respect to the regulatory process as it relates to orphan drugs.
In fact, according to the new study, only eight of the 27 orphan drugs approved between July 2010 and June 2014 were held to "conventional" standards, whereas the rest were approved with some degree of "flexibility," which allows for a case-by-case analysis of each drug and different types of clinical trial data to be used as a basis for approval.